Emerging treatments

Mycophenolate

This works in part by inhibiting the rate-limiting enzyme for the de novo pathway of purine synthesis. Mycophenolate monotherapy and mycophenolate in combination with tacrolimus were found to be ineffective on the relapse rate of nephrotic syndrome in patients with MN.[27][28]​​ However, it has been successfully used in combination with corticosteroids.​​[29][30]​ The general consensus is that mycophenolate has some effect on improving proteinuria in the short term and in the context of small studies, but complete remissions are rare, relapses occur frequently, and long-term benefits are unknown.​​​​[31]

ACTH

Synthetic ACTH given for 1 year decreased proteinuria in patients with MN.​[32]​​[33]​ The mechanism of action of ACTH in this context is unclear; a phase 4 trial to evaluate this effect has been completed.[34]​​ A small randomised pilot study comparing methylprednisolone plus a cytotoxic agent for 6 months versus synthetic ACTH twice weekly for 1 year revealed no significant differences between the 2 groups.​[35]​ Although these studies suggest that prolonged treatment with synthetic ACTH may be effective, more extensive randomised studies with longer follow-up are needed before therapeutic recommendations can be made. The synthetic formulation of ACTH used in these studies is not available in the US.

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